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WO2010048352A3 - Methods for treating eye disorders - Google Patents

Methods for treating eye disorders Download PDF

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Publication number
WO2010048352A3
WO2010048352A3 PCT/US2009/061570 US2009061570W WO2010048352A3 WO 2010048352 A3 WO2010048352 A3 WO 2010048352A3 US 2009061570 W US2009061570 W US 2009061570W WO 2010048352 A3 WO2010048352 A3 WO 2010048352A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
subject
ganglion cell
retinal ganglion
loss
Prior art date
Application number
PCT/US2009/061570
Other languages
French (fr)
Other versions
WO2010048352A2 (en
WO2010048352A8 (en
Inventor
Elena Feinstein
Evgenia Alpert
Igor Mett
Amir Bar-Ilan
Igor Spivak
Hagar Kalinski
Netanja Slager
Original Assignee
Quark Pharmaceuticals, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority to DK09748890.2T priority Critical patent/DK2350279T3/en
Priority to SI200931373T priority patent/SI2350279T1/en
Priority to AU2009308380A priority patent/AU2009308380B2/en
Priority to KR1020117009590A priority patent/KR101672563B1/en
Priority to BRPI0919683-8A priority patent/BRPI0919683A2/en
Priority to US13/062,161 priority patent/US8765931B2/en
Priority to NZ59139109A priority patent/NZ591391A/en
Priority to JP2011533313A priority patent/JP5592892B2/en
Priority to ES09748890.2T priority patent/ES2563984T3/en
Priority to PL09748890T priority patent/PL2350279T3/en
Priority to EA201170551A priority patent/EA022418B1/en
Application filed by Quark Pharmaceuticals, Inc. filed Critical Quark Pharmaceuticals, Inc.
Priority to CN2009801517703A priority patent/CN102449151A/en
Priority to CA2738957A priority patent/CA2738957A1/en
Priority to EP09748890.2A priority patent/EP2350279B1/en
Publication of WO2010048352A2 publication Critical patent/WO2010048352A2/en
Publication of WO2010048352A3 publication Critical patent/WO2010048352A3/en
Priority to IL212986A priority patent/IL212986A/en
Publication of WO2010048352A8 publication Critical patent/WO2010048352A8/en
Priority to US14/217,079 priority patent/US9121020B2/en
Priority to US14/837,366 priority patent/US9701961B2/en
Priority to US15/645,751 priority patent/US20170306329A1/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention relates to compositions and methods for inhibiting loss of a retinal ganglion cell in a subject, comprising non-invasively applying to the surface of the eye of the subject an ophthalmic composition comprising a therapeutically effective amount of at 5 least one siRNA which down regulates expression of a target gene associated with loss of the retinal ganglion cell, thereby inhibiting loss of the retinal ganglion cell in the subject. The methods of the invention also relate to the use of chemically modified siRNA compounds possessing structural motifs which down-regulate the expression of human genes expressed in retinal tissue in the mammalian eye.
PCT/US2009/061570 2008-10-22 2009-10-22 Methods for treating eye disorders WO2010048352A2 (en)

Priority Applications (18)

Application Number Priority Date Filing Date Title
EA201170551A EA022418B1 (en) 2008-10-22 2009-10-22 Methods for treating eye disorders
AU2009308380A AU2009308380B2 (en) 2008-10-22 2009-10-22 Methods for treating eye disorders
KR1020117009590A KR101672563B1 (en) 2008-10-22 2009-10-22 Methods for treating eye disorders
BRPI0919683-8A BRPI0919683A2 (en) 2008-10-22 2009-10-22 METHODS FOR TREATMENT OF DISORDERS
US13/062,161 US8765931B2 (en) 2008-10-22 2009-10-22 Double-stranded oligonucleotide compound for down-regulating the expression of CASP2 gene
NZ59139109A NZ591391A (en) 2008-10-22 2009-10-22 Non-invasive methods for treating eye disorders using a topical oligonucleotide composition
JP2011533313A JP5592892B2 (en) 2008-10-22 2009-10-22 How to treat eye disorders
ES09748890.2T ES2563984T3 (en) 2008-10-22 2009-10-22 Methods for the treatment of eye disorders
CN2009801517703A CN102449151A (en) 2008-10-22 2009-10-22 Methods for treating eye disorders
DK09748890.2T DK2350279T3 (en) 2008-10-22 2009-10-22 METHODS OF TREATING EYE DISEASES
SI200931373T SI2350279T1 (en) 2008-10-22 2009-10-22 Methods for treating eye disorders
PL09748890T PL2350279T3 (en) 2008-10-22 2009-10-22 Methods for treating eye disorders
CA2738957A CA2738957A1 (en) 2008-10-22 2009-10-22 Methods for treating eye disorders
EP09748890.2A EP2350279B1 (en) 2008-10-22 2009-10-22 Methods for treating eye disorders
IL212986A IL212986A (en) 2008-10-22 2011-03-21 Double-stranded oligonucleotide and a pharmaceutical composition
US14/217,079 US9121020B2 (en) 2008-10-22 2014-03-17 Methods for treating eye disorders
US14/837,366 US9701961B2 (en) 2008-10-22 2015-08-27 Methods for treating eye disorders
US15/645,751 US20170306329A1 (en) 2008-10-22 2017-07-10 Methods for treating eye disorders

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US19699508P 2008-10-22 2008-10-22
US61/196,995 2008-10-22
US19893108P 2008-11-11 2008-11-11
US61/198,931 2008-11-11
ILPCT/IL2009/000179 2009-02-15
IL2009000179 2009-02-15

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US13/062,161 A-371-Of-International US8765931B2 (en) 2008-10-22 2009-10-22 Double-stranded oligonucleotide compound for down-regulating the expression of CASP2 gene
US14/217,079 Division US9121020B2 (en) 2008-10-22 2014-03-17 Methods for treating eye disorders

Publications (3)

Publication Number Publication Date
WO2010048352A2 WO2010048352A2 (en) 2010-04-29
WO2010048352A3 true WO2010048352A3 (en) 2010-06-17
WO2010048352A8 WO2010048352A8 (en) 2011-08-11

Family

ID=54839547

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2009/061570 WO2010048352A2 (en) 2008-10-22 2009-10-22 Methods for treating eye disorders

Country Status (15)

Country Link
US (4) US8765931B2 (en)
EP (1) EP2350279B1 (en)
JP (1) JP5592892B2 (en)
KR (1) KR101672563B1 (en)
CN (3) CN102994500B (en)
AU (1) AU2009308380B2 (en)
CA (2) CA2962219C (en)
DK (1) DK2350279T3 (en)
EA (1) EA022418B1 (en)
ES (1) ES2563984T3 (en)
IL (1) IL212986A (en)
NZ (1) NZ591391A (en)
PL (1) PL2350279T3 (en)
SI (1) SI2350279T1 (en)
WO (1) WO2010048352A2 (en)

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